PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]

Sponsor
Nova Laboratories Limited
Study ID
NCT06923111
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
9 Months - 23 Months
Healthy Volunteers
Not accepted

Interventions

  • Xromi — DRUG
    Xromi is indicated for the prevention of vaso-occlusive complications of Sickle Cell Disease in patients over 9 months of age as part of standard clinical practice

Study Details

This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD). The objective is to assess the safety profile and clinical effectiveness of Xromi® under routine clinical conditions. The study includes a prospective cohort of Xromi®-treated patients and a matched retrospective comparator cohort of untreated patients. Participants will be followed for 24 months from treatment initiation or matched index date.

Key Dates

First listed
Apr 11, 2025
Start date
Jun 9, 2025
Status verified
Mar 2026
Primary completion
Jun 30, 2029
Completion
Jun 30, 2029

Study Design

Enrollment
180 participants (estimated)

Arms

  • Arm: Prospective Exposure Cohort
    Children with SCD aged 9 months to under 2 years of age who are newly prescribed Xromi®, will be identified prospectively. These participants will be followed up for 24 months from their index date, regardless of whether they continue treatment with Xromi®, discontinue all hydroxycarbamide treatment, or switch to another formulation of hydroxycarbamide. The decision to prescribe Xromi® will be made solely by the physician independently of the study, as part of standard care. \-
  • Arm: Retrospective Comparator Cohort
    Children with SCD and naïve to any hydroxycarbamide formulation at the index date. These participants will be identified retrospectively using the data from the last 10 years up to the date Xromi® was first used in children from 9 months to under 2 years of age at each individual site. The 24-month follow-up will be retrospective from the date they are matched to the exposed participant, irrespective of whether they start on any formulation of hydroxycarbamide during the follow-up.

Primary Outcome Measure

AESI - Myelosuppression (Neutropenia) [ Time Frame: Pre-baseline, Baseline to 24 months ]

Central Contacts

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